Tag: alzheimer’s

Recon takes an analytical look behind select developments in healthcare

An opinionated take on NEJM highlights for the second quarter of 2024

The quest for solid tumor CAR-T For all their success in hematological cancers, the track record of CAR-Ts against solid tumors has been disappointing (though see here for an exception). One key obstacle has been access by CAR-Ts to the tumor site. Here, a Mass General team reports on their experience treating 3 patients afflicted with glioblastoma multiforme (GBM, a nearly always fatal brain malignancy) positive for a variant of the EGFR receptor. In each case they prepared (autologous) CAR-Ts against the EGFR variant, and delivered them directly into the

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An opinionated take on NEJM highlights for the fourth quarter of 2023

A Chinese study uncovers a new use for an old drug Bleeding in the small intestine is often due to vascular malformations and is difficult to diagnose and manage because 1) the area is hard to reach with a scope and 2) bleeding tends to be intermittent.  Existing approaches are cumbersome and far from satisfactory, which makes a recent blinded RCT study conducted in China exploring thalidomide use in those patients valuable – not only did it show a significant decrease in bleeding episodes after a course of 4 months

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Hearts and souls: An opinionated take on NEJM highlights for October and November 2022

A psychedelic for refractory depression While major depression is awful, treatment-resistant depression (as defined not responding to at least two different courses of therapy) is worse with sufferers stuck in a long dark tunnel of anguish without a light at the end of it. Since psilocybin (the psychedelic agent in so-called magic mushrooms) has shown some effectiveness in depression, it seemed reasonable to test its differentiated mechanism of action in this high need patient population.  In this study that was mostly Europe-based but included sites in the US (finally), patients

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Some very disappointing failures: An opinionated take on NEJM highlights for August and September 2022

Is Parkinson’s α-synuclein going to be as elusive a target as β-amyloid has been for Alzheimer’s? Alpha-synuclein aggregates are characteristic of Parkinson’s disease and genetic variants of this protein clearly lead to familial forms of Parkinson’s; it was therefore reasonable to target alpha-synuclein with antibodies in the hope of modifying disease progression. Sadly, two well designed placebo-controlled studies have now crushed this hope. Both showed absolutely no impact on the progression of patients with early Parkinson’s over times that extended to 1.5 year of follow-up. If there is silver lining,

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Struggling: An opinionated take on NEJM highlights for May 2018

One more on the chin for Alzheimer’s A report of a large phase 3 study of the highly potent oral BACE inhibitor verubecestat (Merck) with yet again a lack of therapeutic effect, despite a dramatic reduction of the cerebrospinal fluid content of beta amyloid in various forms.  With a string of prior failures, this may be the near final blow for amyloid as a treatment target for Alzheimer’s disease (and Biogen’s adacanumab would be the end of the line if it also comes up tails). But note that the study

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